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The phase 2 trial is expected to include 80 adults with genetically confirmed CMT who will be followed for a 21-day treatment period, using changes in functional assessments as the primary outcome.

The professor in residence at UCLA Health discussed the critical considerations when deciding appropriate patients for gene therapies, specifically AAV vectors, in treating muscular dystrophies. [WATCH TIME: 4 minutes]

The SKYCLARYS PASS registry, an international observational cohort study, aims to include approximately 300 omaveloxolone-naïve patients participating in the Friedreich’s Ataxia Global Consortium's UNIFIED natural history study.

The change in opinion was after reconsidering the totality of data from the phase 3 Clarity AD trial, a large-scale study of lecanemab featuring more than 1500 patients with early-stage Alzheimer disease.

The professor in residence at UCLA Health provided clinical insight on the limitations of certain gene therapy vehicles for muscular dystrophies, and the need for better vectors that are more myotropic. [WATCH TIME: 3 minutes]

Investigators are set to test the effects of 300 mg/day fasudil after the therapy showed promising data on ALS-related biomarkers in 180 mg/day doses.

Here's the latest multidisciplinary multiple sclerosis (MS) research from the International Journal of MS Care.

Here's some of what is coming soon to NeurologyLive® this week.

The William S. and Lois Stiles Edgerly Professor of Neurology at Yale School of Medicine talked about the ongoing evolution of multiple sclerosis diagnostic criteria and the importance of comprehensive testing to guide early intervention and improve outcomes. [WATCH TIME: 5 minutes]

A safety analysis of the phase 3 MOXIe Part 2 trial (NCT0225435) highlighted the frequency and timing of TEAEs with omaveloxolone (Skyclarys; Biogen) treatment.

According to the company, VQ-101 is the first small molecule to show over 75% activation of the lysosomal enzyme GCase in humans, with phase 1 results in healthy volunteers indicating tolerability and exposure levels that support once-daily dosing.

Neurology News Network. for the week ending November 9, 2024. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 15, 2024.

After just 6 weeks of treatment, patients showed antibody levels 16 times higher than those in the placebo group, highlighting the vaccine’s potential to slow disease progression.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Lara Jehi, MD, MHCDS. [LISTEN TIME: 20 minutes]

Initial findings showed significant improvements from baseline in the disease-specific, patient-reported quality of life scores in both the high and low dose AAV-GAD groups, with no significant change in the sham group at 26 weeks.

In a previously completed phase 1 study, the treatment resulted in significant improvements in wakefulness at doses of 1.0 mg and 2.5 mg, prompting a follow-up study.

The software, designed using thousands of brain MRI scans, is used for automated detection and severity grading of ARIA-E and ARIA-H for safety monitoring of new Alzheimer disease therapies.

The doctor of nursing practice specialized in movement disorders discussed specific data the StrivePD app captures and how it can enhance quality of life for patients with Parkinson disease. [WATCH TIME: 3 minutes]

Nerivio, a nondrug option for acute migraine attacks and prevention of migraine with or without aura, has been FDA-approved for several years in adolescents aged 12 and older as well as adults.

NGN-401, an AAV9 gene therapy in development for Rett syndrome, was considered well-tolerated, with treated patients showing consistent gains in skill and developmental milestones.

A previous 5-year analysis showed that pediatric patients with aromatic L-amino acid decarboxylase deficiency when treated with eladocagene exuparvovec experienced durable developmental, motor, and cognitive improvements.

The epilepsy specialist and Cleveland Clinic’s Chief Research and Information Officer provided clinical background on a promising study employing a machine learning algorithm to predict post-operative seizure outcomes using a peri-ictal scalp EEG. [WATCH TIME: 6 minutes]

In a longitudinal study lasting 2 years, impaired test performance in semantic fluency and congruent speed were observed in patients with MOGAD relative to healthy controls.

The senior scientist at Sunnybrook Research Institute in Toronto, Ontario, provided clinical insight on the advantages of a flipped classroom, and why neurology educators should consider this approach. [WATCH TIME: 2 minutes]

The doctor of nursing practice specialized in movement disorders provided clinical insight on how the StrivePD application empowers patients with Parkinson disease to track symptoms, medication, and daily activities using an iPhone and Apple Watch. [WATCH TIME: 3 minutes]

The William S. and Lois Stiles Edgerly Professor of Neurology at Yale School of Medicine discussed the newly updated multiple sclerosis criteria, highlighting their focus on probabilistic assessment, advanced technology integration, and early intervention to improve patient care.

The consultant neurologist and senior lecturer at St. Georges Hospital in London, explores the origins of common neuropsychiatric symptoms experienced by patients with Parkinson disease and how they may be exasperated. [WATCH TIME: 4 minutes]